Naphthyridine-azaquinolone (NA) is a specific small molecule that specifically binds slipped-CAG DNA intermediates of expansion mutations.
Naphthyridine-azaquinolone specifically inhibits repair of long CAG slip-outs by human (HeLa) cell extracts.
Naphthyridine-azaquinolone modulates instability of expanded mutant repeats in Huntington's disease (HD) cells, with limited off-target effects.
NA induces CAG contractions, independent of proliferation, dependent on rCAG transcription, affects interaction of DNA repair proteins on long CAG slip-outs.
Naphthyridine-azaquinolone modulates repeat instability in the striatum of HD mice, induces a reduction in mHTT aggregates in R6/2 mice.
Naphthyridine-azaquinolone-induced contractions depend upon active expansions driven by MutSβ.
